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Role of Lipid-Based and Polymer-Based Non-Viral Vectors in Nucleic Acid Delivery for Next-Generation Gene Therapy

The field of gene therapy has experienced an insurgence of attention for its widespread ability to regulate gene expression by targeting genomic DNA, messenger RNA, microRNA, and short-interfering RNA for treating malignant and non-malignant disorders. Numerous nucleic acid analogs have been develop...

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Autores principales: Wahane, Aniket, Waghmode, Akaash, Kapphahn, Alexander, Dhuri, Karishma, Gupta, Anisha, Bahal, Raman
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7356024/
https://www.ncbi.nlm.nih.gov/pubmed/32580326
http://dx.doi.org/10.3390/molecules25122866
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author Wahane, Aniket
Waghmode, Akaash
Kapphahn, Alexander
Dhuri, Karishma
Gupta, Anisha
Bahal, Raman
author_facet Wahane, Aniket
Waghmode, Akaash
Kapphahn, Alexander
Dhuri, Karishma
Gupta, Anisha
Bahal, Raman
author_sort Wahane, Aniket
collection PubMed
description The field of gene therapy has experienced an insurgence of attention for its widespread ability to regulate gene expression by targeting genomic DNA, messenger RNA, microRNA, and short-interfering RNA for treating malignant and non-malignant disorders. Numerous nucleic acid analogs have been developed to target coding or non-coding sequences of the human genome for gene regulation. However, broader clinical applications of nucleic acid analogs have been limited due to their poor cell or organ-specific delivery. To resolve these issues, non-viral vectors based on nanoparticles, liposomes, and polyplexes have been developed to date. This review is centered on non-viral vectors mainly comprising of cationic lipids and polymers for nucleic acid-based delivery for numerous gene therapy-based applications.
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spelling pubmed-73560242020-07-22 Role of Lipid-Based and Polymer-Based Non-Viral Vectors in Nucleic Acid Delivery for Next-Generation Gene Therapy Wahane, Aniket Waghmode, Akaash Kapphahn, Alexander Dhuri, Karishma Gupta, Anisha Bahal, Raman Molecules Review The field of gene therapy has experienced an insurgence of attention for its widespread ability to regulate gene expression by targeting genomic DNA, messenger RNA, microRNA, and short-interfering RNA for treating malignant and non-malignant disorders. Numerous nucleic acid analogs have been developed to target coding or non-coding sequences of the human genome for gene regulation. However, broader clinical applications of nucleic acid analogs have been limited due to their poor cell or organ-specific delivery. To resolve these issues, non-viral vectors based on nanoparticles, liposomes, and polyplexes have been developed to date. This review is centered on non-viral vectors mainly comprising of cationic lipids and polymers for nucleic acid-based delivery for numerous gene therapy-based applications. MDPI 2020-06-22 /pmc/articles/PMC7356024/ /pubmed/32580326 http://dx.doi.org/10.3390/molecules25122866 Text en © 2020 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Wahane, Aniket
Waghmode, Akaash
Kapphahn, Alexander
Dhuri, Karishma
Gupta, Anisha
Bahal, Raman
Role of Lipid-Based and Polymer-Based Non-Viral Vectors in Nucleic Acid Delivery for Next-Generation Gene Therapy
title Role of Lipid-Based and Polymer-Based Non-Viral Vectors in Nucleic Acid Delivery for Next-Generation Gene Therapy
title_full Role of Lipid-Based and Polymer-Based Non-Viral Vectors in Nucleic Acid Delivery for Next-Generation Gene Therapy
title_fullStr Role of Lipid-Based and Polymer-Based Non-Viral Vectors in Nucleic Acid Delivery for Next-Generation Gene Therapy
title_full_unstemmed Role of Lipid-Based and Polymer-Based Non-Viral Vectors in Nucleic Acid Delivery for Next-Generation Gene Therapy
title_short Role of Lipid-Based and Polymer-Based Non-Viral Vectors in Nucleic Acid Delivery for Next-Generation Gene Therapy
title_sort role of lipid-based and polymer-based non-viral vectors in nucleic acid delivery for next-generation gene therapy
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7356024/
https://www.ncbi.nlm.nih.gov/pubmed/32580326
http://dx.doi.org/10.3390/molecules25122866
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